Distal Myopathy Nonaka Type
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS trial_result · 2019-02-19Clinical Trial Update: Study to Evaluate the Safety and Efficacy of Aceneuramic Acid Extended-Release (Ace-ER) Tablets in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM) [Phase 3]
Phase 3 trial evaluating Ace-ER in GNEM/HIBM was terminated; results were posted in 2019.
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CLINICAL_TRIALS trial_result · 2018-07-09Clinical Trial Update: Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Sialic Acid in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM) [Phase 3]
A Phase 3 randomized, double-blind, placebo-controlled study evaluating sialic acid in GNEM/HIBM (GNE myopathy) has completed with results p…